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Producing transgenic mammal comprising reduced expression of DNAse I, useful for studying development of systemic lupus erythematosus and in drug screening

机译:生产包含降低的DNAse I表达的转基因哺乳动物,可用于研究系统性红斑狼疮的发展和药物筛选

摘要

Producing, (P), a transgenic, non-human mammal (A) that has at least partial reduction in activity of DNAse I (I), is new. Production of a transgenic, non-human mammal (A) that has at least partial reduction in activity of DNAse I (I), where an embryonal stem cell is transfected with a (I)-targeting vector and stable clones isolated in which the endogenous (I)-encoding gene has been (partially) replaced by other sequences or destroyed, as a result of homologous recombination with parts of the targeting vector. These clones are implanted into a suitably prepared surrogate dam and progeny selected for presence of DNA incorporation. Independent claims are also included for the following: (1) (A) produced by P; (2) recombinant (I)-targeting vector used for homologous recombination; (3) stably transfected cells in which all or part of the (I)-encoding gene has been replaced by another DNA sequence; (4) model system for analysis of either the origins of SLE (systemic lupus erythematosus) or treatments of diseases, specifically SLE, that includes at least one (A); (5) testing new pharmaceuticals for activity against SLE, using (A) or stable cell clones or organs derived from (A); and (6) testing the affects of genetic, biological, chemical and physical factors on development of SLE.
机译:生产(P)至少部分降低了DNAse I(I)活性的转基因非人类哺乳动物(A)是新的。产生至少部分降低DNAse I(I)活性的转基因非人类哺乳动物(A),其中将胚胎干细胞用(I)靶向载体转染,并分离出稳定的克隆,其中内源性(I)编码基因已经(部分)被其他序列取代或被破坏,这是由于与目标载体部分进行了同源重组的结果。将这些克隆植入适当制备的替代坝中,并根据DNA掺入情况选择后代。还包括以下方面的独立权利要求:(1)(A)由P提出; (2)用于同源重组的重组(I)-靶向载体; (3)稳定转染的细胞,其中(I)编码基因的全部或部分已被另一DNA序列取代; (4)用于分析系统性红斑狼疮(系统性红斑狼疮)起源或疾病治疗的模型系统,特别是系统性红斑狼疮,包括至少一个(A); (5)使用(A)或衍生自(A)的稳定细胞克隆或器官测试新药对SLE的活性; (6)检验遗传,生物学,化学和物理因素对SLE发展的影响。

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